US FDA approves Italfarmaco's drug for Duchenne muscle-wasting disorder

By Mariam Sunny and Sruthi Narasimha Chari

(Reuters) – The U.S. FDA has approved privately held Italfarmaco Group’s drug to treat Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disorder, the health regulator said on Thursday.

The oral drug, to be sold under the brand name Duvyzat, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD, according to the FDA.

Duvyzat has been approved for patients six years of age and older.

DMD affects an estimated one-in-3,500 male births worldwide, and most patients lack the protein dystrophin which keeps muscles intact.

Duvyzat, chemically known as givinostat, helps regulate a group of enzymes responsible for causing muscle damage and deterioration in DMD patients.

Italfarmaco’s application for the drug was based on data from a late-stage study conducted on 120 patients on chronic steroids, in which the drug helped slow disease progression in ambulant boys with DMD aged 6 years and above after 18 months of treatment.

The approval pits the drug against already approved DMD treatments in the market, including Sarepta Therapeutics’ bestselling medicine Exondys 51, as well as its other therapies Vyondys and Amondys.

These belong to a class of “exon-skipping” therapies, which work by skipping specific parts of genes, called exons, helping the body make shorter forms of dystrophin.

Unlike other available treatments, Duvyzat tries to “counterbalance” the effects caused by a lack of dystrophin, Italfarmaco Chief Medical Officer Paolo Bettica said ahead of the decision.

“When you give givinostat, you have less muscle damage, less inflammation, less fibrosis and fat replacement, and an increase in muscle regeneration,” Bettica added.

The FDA, last year, approved Sarepta’s first-of-its-kind gene therapy for DMD patients aged between 4 and 5 years who can walk, based on trial data that showed it was able to produce a mini version of the dystrophin protein.

The therapy, Elevidys, is one of the world’s most expensive treatments with a wholesale cost of $3.2 million per patient.

(Reporting by Sruthi Narasimha Chari and Mariam Sunny in Bengaluru; Editing by Krishna Chandra Eluri)

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